Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as lufepirsen high dose
Lufepirsen · 1 trial · 1 indication
The proportion of subjects achieving corneal re-epithelialization that is maintained for a minimum of 28 days, based on assessment of corneal fluorescein staining images of the PCED by a Central Reading Center (CRC).
| Arm | Type | Description |
|---|---|---|
| NEXAGON® (lufepirsen ophthalmic gel) High Dose Concentration | EXPERIMENTAL | Lufepirsen (High dose concentration) applied topically to the eye once a week for a minimum of 4 weeks to a maximum of 8 weeks. |
| NEXAGON Vehicle (ophthalmic gel) | PLACEBO_COMPARATOR | Vehicle applied topically to the eye once a week for a minimum of 4 weeks to a maximum of 8 weeks. |
| NEXAGON® (lufepirsen ophthalmic gel) Low Dose Concentration (EU sites only) | EXPERIMENTAL | Lufepirsen (Low dose concentration) applied topically to the eye once a week for a minimum of 4 weeks to a maximum of 8 weeks. (EU sites only). |
| Name | Type | Description |
|---|---|---|
| lufepirsen high dose | DRUG | Lufepirsen is an unmodified connexin43 antisense oligonucleotide. |
| Vehicle | DRUG | Matching vehicle without lufepirsen. |
| lufepirsen low dose | DRUG | Lufepirsen is an unmodified connexin43 antisense oligonucleotide. |
Inclusion Criteria: 1. Have a PCED that is at least 2 weeks in duration and refractory to one or more conventional non-surgical standard of care (SOC) treatments 2. Have no clinical evidence of improvement in the PCED within 2 weeks prior to randomization despite the use of non-surgical SOC treatme...
Lufepirsen is an investigational RNA therapy being developed by Glaukos Corporation for persistent corneal epithelial defect, an eye condition in which the cornea's outer surface fails to heal. It is given as an ophthalmic gel and is currently in Phase 2 clinical development. It is not yet approved for commercial use.
Lufepirsen is being studied as a treatment for persistent corneal epithelial defect, a condition where the corneal surface does not heal after injury or disease. The Phase 2 trial evaluates whether the ophthalmic gel can improve corneal healing in affected patients, including children as young as 2 years old.
Lufepirsen targets GJA1, the gene encoding connexin 43. It is an antisense oligonucleotide, part of the -sen drug class, designed to reduce expression of that target. In persistent corneal epithelial defect, this mechanism is intended to support corneal wound healing, though the approach remains investigational.
Glaukos Corporation, which trades on the New York Stock Exchange under the ticker GKOS, is developing lufepirsen. The company is running the Phase 2 program for the ophthalmic gel formulation in persistent corneal epithelial defect across sites in the United States and Europe.
Lufepirsen is in Phase 2 clinical development. It is an investigational therapy and has not been approved by the FDA or any other regulatory agency. The ongoing Phase 2 study is randomized, double blind, and placebo controlled, and it is currently recruiting participants.
Lufepirsen is being evaluated in one active clinical trial, NCT05966493, titled a study to evaluate the safety and efficacy of NEXAGON (lufepirsen ophthalmic gel) in subjects with PCED. The Phase 2 trial is recruiting 84 participants across the United States, Germany, Italy, and Spain.
Lufepirsen high dose is an alternative name used for lufepirsen, so both terms refer to the same investigational Glaukos RNA therapy. The Phase 2 trial is placebo controlled and randomized, and it enrolls 84 participants with persistent corneal epithelial defect across four countries.